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CYCLIC COMPOUNDS USED AS MULTI-TARGET KINASE INHIBITORS AND PREPARATION METHOD THEREFOR

NºPublicación:  EP4620959A1 24/09/2025
Solicitante: 
SHANGHAI INST ORGANIC CHEMISTRY CAS [CN]
Shanghai Institute of Organic Chemistry, Chinese Academy of Sciences
EP_4620959_PA

Resumen de: EP4620959A1

The present invention discloses cyclic compounds as multi-target kinase inhibitors and preparation methods thereof. The multi-target kinase inhibitors of the present invention are as shown in general formula I, wherein R<sup>1</sup>, R<sup>2</sup>, R<sup>3</sup>, R<sup>3a</sup>, L<sup>1</sup>, L<sup>2</sup>, L<sup>3</sup>, ring A, and ring B are as shown in the Specification and Claims. The present invention also discloses preparation methods of general formula I and its inhibitory activity against multiple kinases. The compounds of general formula I described in the present invention can be used for treating cancers and neurodegenerative diseases such as Parkinson's disease, etc.

TREATING AMYOTROPHIC LATERAL SCLEROSIS WITH PRIDOPIDINE

NºPublicación:  EP4620471A2 24/09/2025
Solicitante: 
PRILENIA NEUROTHERAPEUTICS LTD [IL]
Prilenia Neurotherapeutics Ltd
EP_4620471_A2

Resumen de: EP4620471A2

Pridopidine or a pharmaceutically acceptable salt thereof for use in treating a subject afflicted with amyotrophic lateral sclerosis (ALS).

HUMANISED ANTIBODY AGAINST AMYLOID BETA 42

NºPublicación:  AU2024238598A1 18/09/2025
Solicitante: 
ALZINOVA AB
ALZINOVA AB
AU_2024238598_A1

Resumen de: AU2024238598A1

The present invention provides a humanised antibody comprising an antigen-binding domain capable of binding specifically to Aβ42 prefibrillar oligomers with β structure, said antigen-binding domain comprising: (iii) a heavy chain variable region (VH) comprising the sequence of SEQ ID NO.1; or (iv) a light chain variable region (VL) comprising the sequence of SEQ ID NO.2; or a combination thereof. Also provided is the use of the antibody in the treatment of an amyloid disease, and particularly Alzheimer's disease, conjugates and pharmaceutical compositions comprising the antibody, and nucleic acid molecules encoding the antibody or a heavy or light chain polypeptide thereof, as well as vectors and host cells comprising such a molecule.

TREATMENT OF PARKINSON'S DISEASE IN A PATIENT USING A GLUCOCEREBROSIDASE ACTIVATOR

NºPublicación:  AU2024237252A1 18/09/2025
Solicitante: 
BIAL - R&D INVESTMENTS S A
BIAL - R&D INVESTMENTS, S.A
AU_2024237252_A1

Resumen de: AU2024237252A1

Methods for preventing, limiting or delaying clinical motor progression in a subject with Parkinson's disease with low GCase activity, such as a PD patient with a pathogenic variant in the glucocerebrosidase 1 (GBA1) gene (GBA-PD) is provided, said methods comprising administering a therapeutically effective amount of 5,7-dimethyl-N-((1R,4R)-4- (pentyloxy)cyclohexyl)pyrazolol1,5-apyrimidine-3-carboxamide (Compound A), or a pharmaceutically acceptable salt thereof, to said subject.

PEPTIDE TO TREAT ALPHA-SYNUCLEIN AMYLOID BASED DISORDERS

NºPublicación:  US2025289849A1 18/09/2025
Solicitante: 
COUNCIL SCIENT IND RES [IN]
COUNCIL OF SCIENTIFIC & INDUSTRIAL RESEARCH
US_2025289849_PA

Resumen de: US2025289849A1

The present invention relates to a cell-penetrating peptide and its derivatives to inhibit α-synuclein fibrillation. The present invention specifically relates to a peptide-based inhibitor of Parkinson's Disease. The present invention discloses the identification of the peptides permeable to blood brain barrier for inhibition. α-synuclein fibril formation is observed in the presence of peptides as indicated by SEQ. ID-7, SEQ. ID-8 wherein the SEQ. ID-7, SEQ. ID-8 are truncated versions of SEQ. ID-4 having the homology of 83% and 75% respectively. It provides a peptide having an amino acid sequence of Formula 1. The present invention also provides a pharmaceutical composition comprising a peptide of Formula 1 along with the pharmaceutically acceptable excipient(s) having inhibitory activity against β-sheet polymerisation of amyloidogenic proteins. The analysis showed that peptides corresponding to SEQ. ID-7, SEQ. ID-8 are better inhibitors than SEQ. ID-4 against α-synuclein fibrillation.

SELECTIVE LIGANDS FOR TAU AGGREGATES

NºPublicación:  US2025289803A1 18/09/2025
Solicitante: 
SENTONIX INC [US]
Sentonix, Inc
US_2025289803_PA

Resumen de: US2025289803A1

The invention provides a compound of formula (I), or a pharmaceutically acceptable salt, ester or carbamate thereof, or a salt of such an ester or carbamate,wherein either:R1 is OH, and R2 is H; orR1 is H, and R2 is OH.The invention further provides uses of the compounds of formula (I) and compositions comprising compounds of formula (I), including the use of such compounds for the detection of tau deposits, and the use of such compounds and compositions as diagnostic agents in the diagnosis or monitoring of the progression of a disease or disorder such as Alzheimer's disease, progressive supranuclear palsy and corticobasal degeneration, or for the prevention or treatment of a disease or disorder such as Alzheimer's disease, progressive supranuclear palsy and corticobasal degeneration.

VARIANT RNAi

NºPublicación:  US2025290075A1 18/09/2025
Solicitante: 
GENZYME CORP [US]
Genzyme Corporation
US_2025290075_PA

Resumen de: US2025290075A1

Provided herein are RNAi molecules for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.

PATHOLOGY-RESPONSIVE RECOMBINANT CELLS AND USES THEREOF

NºPublicación:  US2025290037A1 18/09/2025
Solicitante: 
UNIV CALIFORNIA [US]
THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
US_2025290037_PA

Resumen de: US2025290037A1

Modified cells that express and present or secrete at least one therapeutic molecule that can treat or ameliorate a disease of interest such as but not limited to Alzheimer's disease. In the modified cells, expression of the therapeutic molecule is induced when the modified cells are proximate to or in contact with pathology related to the disease of interest. The present disclosure also relates to compositions and kits comprising the disclosed cells. The present disclosure also relates to methods of using the disclosed cells for treating disease.

Pulsatile drug delivery system for treating morning akinesia

NºPublicación:  AU2025223847A1 18/09/2025
Solicitante: 
CONTERA PHARMA AS
Contera Pharma A/S
AU_2025223847_A1

Resumen de: AU2025223847A1

Abstract Provided herewith is a pharmaceutical composition comprising, separately or together, a pulsatile release component comprising levodopa and a DOPA decarboxylase inhibitor for the management of OFF-time episodes in patients with Parkinson’s disease. 5 Abstract Provided herewith is a pharmaceutical composition comprising, separately or together, a pulsatile release component comprising levodopa and a DOPA decarboxylase inhibitor for the management of OFF-time episodes in patients with Parkinson's 5 disease. ug b s t r a c t r o v i d e d h e r e w i t h i s a p h a r m a c e u t i c a l c o m p o s i t i o n c o m p r i s i n g , s e p a r a t e l y o r t o g e t h e r , u g a p u l s a t i l e r e l e a s e c o m p o n e n t c o m p r i s i n g l e v o d o p a a n d a d e c a r b o x y l a s e i n h i b i t o r f o r t h e m a n a g e m e n t o f - t i m e e p i s o d e s i n p a t i e n t s w i t h a r k i n s o n ' s d i s e a s e

BAG3 AND PROTEIN QUALITY CONTROL IN THE BRAIN

NºPublicación:  US2025289858A1 18/09/2025
Solicitante: 
UNIV TEMPLE [US]
TEMPLE UNIVERSITY OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION
US_2025289858_A1

Resumen de: US2025289858A1

BAG3 protein is used in the treatment of, for example, is Parkinson's disease, Alzheimer's disease, Amyotrophic Lateral Sclerosis, Huntington disease, Lewy body disease, vascular dementia, mixed dementia and Traumatic Brain Injury, for example, complicated by Chronic Traumatic Encephalopathy (CTE). Therapeutically effective BAG3 compositions, BAG3 uses and BAG3 methods of treatment are described.

DELIVERY OF TAU REPRESSORS USING THE BLOOD-BRAIN BARRIER PENETRANT CAPSID

NºPublicación:  WO2025193848A1 18/09/2025
Solicitante: 
SANGAMO THERAPEUTICS INC [US]
SANGAMO THERAPEUTICS, INC
WO_2025193848_PA

Resumen de: WO2025193848A1

Provided are compositions for delivery of zinc finger fusion proteins that inhibit expression of tau in the nervous system using the blood-brain barrier penetrant AAV capsid proteins comprising SEQ ID. NO: 1235, and methods of using the compositions to treat neurodegenerative diseases such as Alzheimer's disease, frontotemporal dementia, and other tauopathies.

A TRISOMY LINKED HEMATOPOIETIC GENE VARIANT FOR TREATING ALZHEIMER'S DISEASE

NºPublicación:  WO2025193743A1 18/09/2025
Solicitante: 
UNIV RUTGERS [US]
RUTGERS, THE STATE UNIVERSITY OF NEW JERSEY
WO_2025193743_PA

Resumen de: WO2025193743A1

Provided herein are systems and methods for introducing an A455D mutation into CSF2RB to neurodegenerative diseases, such as Alzheimer's disease.

PHARMACEUTICAL COMPOSITION FOR PREVENTING OR TREATING ALZHEIMER'S DISEASE, COMPRISING NEURAL CREST-DERIVED NASAL TURBINATE STEM CELLS EXPRESSING SSEA3 AND CD105 AS ACTIVE INGREDIENT

NºPublicación:  WO2025192800A1 18/09/2025
Solicitante: 
CATHOLIC UNIV KOREA IND ACADEMIC COOPERATION FOUNDATION [KR]
\uAC00\uD1A8\uB9AD\uB300\uD559\uAD50 \uC0B0\uD559\uD611\uB825\uB2E8
WO_2025192800_A1

Resumen de: WO2025192800A1

The present invention relates to a pharmaceutical composition for preventing or treating Alzheimer's disease, the composition comprising, as an active ingredient, neural crest-derived nasal turbinate stem cells (NTSCs) expressing SSEA3 and CD105. Treatment with the NTSCs expressing SSEA3 and CD105 or with an NTSC cell line including at least a predetermined proportion of the NTSCs was found to result in remarkably good therapeutic activity against Alzheimer's disease. Therefore, the present invention is expected to be effectively used not only as a composition for preventing or treating Alzheimer's disease in which the composition includes, as an active ingredient, NTSCs expressing SSEA3 and CD105 or an NTSC cell line including at least a predetermined proportion of the NTSCs, but also for uses such as screening of NTSC formulations that can be used to treat Alzheimer's disease, or prediction of the therapeutic efficacy thereof.

AUTOPHAGY ENHANCERS

NºPublicación:  WO2025194036A1 18/09/2025
Solicitante: 
WASHINGTON UNIVERSITY ST LOUIS [US]
WASHINGTON UNIVERSITY
WO_2025194036_PA

Resumen de: WO2025194036A1

The present disclosure is generally directed to tetracyclic analogs that modulate autophagy in a subject suffering from alpha-1 antitrypsin deficiency (ATD) and possibly other autophagy associated diseases or disorders, such as Alzheimer's disease, Parkinson's disease, Huntington's disease, and amyotrophic lateral sclerosis.

REAGENT, PHARMACEUTICAL COMPOSITION AND METHODS FOR DETECTING, EVALUATING THE PROGRESSION, AND TREATING DISEASES ASSOCIATED WITH B-AMYLOID PLAQUES

NºPublicación:  US2025288701A1 18/09/2025
Solicitante: 
UNIV CHILE [CL]
UNIV PONTIFICIA CATOLICA CHILE [CL]
UNIVERSIDAD DE CHILE,
PONTIFICIA UNIVERSIDAD CAT\u00D3LICA DE CHILE
US_2025288701_PA

Resumen de: US2025288701A1

The present invention relates to the technical field of nanotechnology and pharmaceutics, particularly, it relates to a reagent and pharmaceutical composition containing gold nanorods conjugated to D1 and Angiopep-2 peptides, and methods for detecting, diagnosing, evaluating the progression, and treating diseases related to β-amyloid plaques, such as Alzheimer's disease. Surprisingly, with the reagent and pharmaceutical composition of the present invention, a dose of gold per kg of body weight well below those previously reported is required, hence their applications are more economical, in addition to decreasing the likelihood of toxic effects, thanks to the low doses required to exert their function.

INFLAMMATORY CYTOKINE PRODUCTION INHIBITOR

NºPublicación:  US2025288615A1 18/09/2025
Solicitante: 
SAISEI PHARMA CO LTD [JP]
SAISEI PHARMA CO., LTD
US_2025288615_A1

Resumen de: US2025288615A1

Provided are inflammatory cytokine production inhibitors which inhibit the production of the inflammatory cytokines TNF-α and IL-1β and can be applied to drugs effective against inflammatory diseases caused by overproduction of these inflammatory cytokines, such as chronic inflammation (e.g., rheumatoid arthritis, ulcerative colitis) and Crohn's disease, as well as type 2 diabetes, depression, obesity, sepsis, atherosclerosis, dermatitis, dementia, schizophrenia, and Parkinson's disease, and also to foods and drinks such as health foods. The inflammatory cytokine production inhibitors contain as an active ingredient an enzyme-treated whey obtained by contacting whey with β-galactosidase. The enzyme-treated whey is obtained by further contacting with sialidase.

TREATMENTS FOR AMYOTROPHIC LATERAL SCLEROSIS USING DAZUCORILANT

NºPublicación:  US2025288580A1 18/09/2025
Solicitante: 
CORCEPT THERAPEUTICS INC [US]
Corcept Therapeutics Incorporated
US_2025288580_PA

Resumen de: US2025288580A1

Applicant discloses methods and compositions for treating a patient suffering from amyotrophic lateral sclerosis (ALS) comprising administration of a heteroaryl ketone fused azadecalin compound. In embodiments, the heteroaryl ketone fused azadecalin compound is dazucorilant: (R)-(1-(4-fluorophenyl)-6-((4-(trifluoromethyl)phenyl) sulfonyl)-4,4a,5,6,7,8-hexahydro-1-H-pyrazolo3,4-gisoquinolin-4a-yl) (pyridin-2-yl)methanone, having the chemical structure illustrated asSuitable doses include daily administration of 150 milligrams and 300 milligrams of dazucorilant. Suitable doses include daily administration of dazucorilant with food, or with water, or with food and water. Daily administration of dazucorilant is effective to increase dazucorilant exposure up to about 2-fold when continued for seven days or more. Administration of such a heteroaryl ketone fused azadecalin compound may comprise oral administration, enteral administration, or other administration. Pharmaceutical compositions comprising dazucorilant are useful in the treatment of patients suffering from ALS. Suitable pharmaceutical compositions comprising dazucorilant include, e.g., pharmaceutical compositions for oral administration and pharmaceutical compositions for enteral administration.

CRYSTAL FORMS OF TETRAHYDRO-N,N-DIMETHYL-2,2-DIPHENYL-3-FURANMETHANAMINE HYDROCHLORIDE, PROCESSES OF MAKING SUCH FORMS, AND THEIR PHARMACEUTICAL COMPOSITIONS

NºPublicación:  EP4616852A2 17/09/2025
Solicitante: 
ANAVEX LIFE SCIENCES CORP [US]
Anavex Life Sciences Corp
EP_4616852_PA

Resumen de: EP4616852A2

Polymorphic forms of tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanmethanamine hydrochloride (ANAVEX2-73) and a metabolite of tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanmethanaminehydrochloride (ANAVEX2-73) are disclosed and characterized. Compositions and method for treatment of Alzheimer's disease that includes the polymorphic forms and metabolite of tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanmethanamine hydrochloride (ANAVEX2-73).

TREM2 ANTIGEN BINDING PROTEINS AND USES THEREOF

NºPublicación:  EP4617286A2 17/09/2025
Solicitante: 
AMGEN INC [US]
Amgen Inc
EP_4617286_PA

Resumen de: EP4617286A2

The present invention relates to antigen binding proteins, such as monoclonal antibodies, that specifically bind to and activate human triggering receptor expressed on myeloid cells-2 (TREM2) and pharmaceutical compositions comprising such antigen binding proteins. The agonist antigen binding proteins (e.g. antibodies) of the invention are capable of activating TREM2/DAP12 signaling in myeloid cells in the absence of Fe-mediated crosslinking of the antigen binding proteins. Methods of treating or preventing conditions associated with TREM2 loss of function, such as Alzheimer's disease and multiple sclerosis, using the antigen binding proteins are also described.

PEPTIDE FOR TREATMENT OF COGNITIVE DISEASES

NºPublicación:  WO2025186220A1 12/09/2025
Solicitante: 
PRIAVOID GMBH [DE]
PRIAVOID GMBH
WO_2025186220_PA

Resumen de: WO2025186220A1

The present invention relates to peptides for treatment of cognitive diseases, in particular Alzeheimer's Disease, mild cognitive impairment due to Alzheimer's disease, and mild dementia due to Alzheimer's disease.

VECTORIZED ANTI-TDP-43 ANTIBODIES

NºPublicación:  WO2025186332A1 12/09/2025
Solicitante: 
AC IMMUNE SA [CH]
AC IMMUNE SA
WO_2025186332_A1

Resumen de: WO2025186332A1

The present invention is in the field of AAV vectorized TDP-43 specific antibodies and uses thereof. The present invention further relates to means and methods to prevent, alleviate and/or treat a disease, disorder and/or abnormality associated with TDP-43, or TDP-43 proteinopathy, including but not limited to Frontotemporal dementia (FTD), amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), Chronic Traumatic Encephalopathy (CTE), and limbic-predominant age-related TDP-43 encephalopathy (LATE).

ANTI-GALECTIN 3 ANTIBODIES AND THEIR USE IN EPILEPSY AND RELATED DISEASES

NºPublicación:  WO2025185724A1 12/09/2025
Solicitante: 
SUNMED THERAPEUTIC LTD [CN]
SUNMED THERAPEUTIC LIMITED
WO_2025185724_PA

Resumen de: WO2025185724A1

Provided herein are antibodies that target Galectin-3. Such antibodies are used in methods of treating epilepsy and related neurological disorders, such as Alzheimer's disease (AD) and Parkinson's disease (PD).

METHODS OF TREATING PARKINSON'S DISEASE WITH T-TYPE CALCIUM CHANNEL MODULATORS

NºPublicación:  WO2025188619A1 12/09/2025
Solicitante: 
PRAXIS PREC MEDICINES INC [US]
PRAXIS PRECISION MEDICINES, INC
WO_2025188619_PA

Resumen de: WO2025188619A1

Described herein, in part, are methods useful for preventing and/or treating a disease or condition relating to aberrant function or activity of a T-type calcium channel, such as Parkinson's disease, psychiatric disorders (e.g., mood disorder (e.g., major depressive disorder)), pain, tremor (e.g., essential tremor), seizures (e.g., absence seizures), epilepsy, or an epilepsy syndrome (e.g., juvenile myoclonic epilepsy). The present invention further comprises methods for modulating the function of a T-type calcium channel and methods of administering a titrated dosage of a T-type calcium channel antagonist.

METHODS OF USE OF (4R,5R)-5-(2-CHLOROPHENYL)-4-(5-(PHENYLETHYNYL)PYRIDIN-3-YL)OXAZOLIDIN-2-ONE

NºPublicación:  WO2025188734A1 12/09/2025
Solicitante: 
ALLYX THERAPEUTICS INC [US]
ALLYX THERAPEUTICS, INC
WO_2025188734_PA

Resumen de: WO2025188734A1

The present disclosure provides methods of treating Alzheimer's disease and other disorders using (4R,5R)-5-(2-chlorophenyl)-4-(5-(phenylethynyl)pyridin-3-yl)oxazolidin-2-one (Compound 1).

COMPOSITIONS FOR TARGETING FLI-1 AND METHODS OF USE THEREOF

Nº publicación: US2025283075A1 11/09/2025

Solicitante:

MUSC FOUNDATION FOR RES DEVELOPMENT D/B/A ZUCKER INSTITUTE FOR INNOVATION COMMERCIALIZATION [US]
MUSC FOUNDATION FOR RES DEVELOPMENT [US]
MUSC Foundation for Research Development d/b/a Zucker Institute for Innovation Commercialization,
MUSC FOUNDATION FOR RESEARCH DEVELOPMENT

WO_2023028605_A1

Resumen de: US2025283075A1

The present disclosure provides methods for treating inflammatory disease, such as Alzheimer's disease, by administering an inhibitor of Fli-1, such as an antisense oligonucleotide. Further provided are antisense gapmer oligonucleotides for targeting Fli-1.

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